Drug Type Gene therapy |
Synonyms- |
Target |
Action stimulants |
Mechanism RPE65 stimulants(Retinoid isomerohydrolase stimulants), Gene transference(Gene transference) |
Therapeutic Areas |
Active Indication- |
Inactive Indication |
Originator Organization |
Active Organization- |
Inactive Organization |
License Organization- |
Drug Highest PhasePendingPhase 2 |
First Approval Date- |
Regulation- |
| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Leber Congenital Amaurosis | Phase 2 | United States | 01 Apr 2016 | |
| Leber Congenital Amaurosis | Phase 2 | United Kingdom | 01 Apr 2016 |





